Preliminary data showing an Amylyx Pharmaceuticals drug as a promising new therapy in slowing the progression of ALS was published in The Wall Street Journal Today:

“The drug’s maker, closely held Amylyx Pharmaceuticals Inc., is releasing only the barest outlines of the mid-stage trial for now, as researchers continue to analyze the results. The drug slowed the rate of ALS’s progression in patients as measured by a widely used scale, said the researchers and the company’s founders.

‘We’re just another step closer to hopefully stopping this illness,” said Merit Cudkowicz, who heads the Healey Center for ALS at Massachusetts General Hospital in Boston and helped design and coordinate the study.’”  See The Wall Street Journal article HERE.

The ALS Center of Excellence at Michigan Medicine is currently one of the research sites gathering clinical data.

While the trial is ongoing, it is no longer enrolling new participants.

Stephen Goutman, M.D.

“Our research at the ALS Center of Excellence over the past few years has been building to this detailed examination of the gene-time-environment model,” said Dr. Goutman, who is a U-M associate professor of neurology. “Every person is impacted differently by the environment based on their genes and how their body metabolizes pollutants. We hypothesize that people who have both higher ALS genetic risk and environmental exposures will be shown to have a higher risk of developing ALS and a faster progression of the disease.”

The grant will be co-directed by Kelly Bakulski, Ph.D., U-M assistant professor of epidemiology. By understanding how exposure to pollutants like pesticides and industrial chemicals affects a person’s genes, Drs. Goutman and Bakulski plan to develop new targets for drug treatments. Additionally, their research will likely identify life changes that people can make to reduce their susceptibility to ALS.

Kelly Bakulski, Ph.D.

“Dr. Goutman and I are passionate about defining the links between ALS, the environment and genetics,” said Eva Feldman, M.D., Ph.D., director of the U-M ALS Center of Excellence. “Thanks to the research partnership we have with our patients in the Multidisciplinary ALS Clinic, we already have a great deal of information stored in our biorepository that will help with this new study. I am very excited to see what the results will tell us about why Michigan has the highest prevalence of ALS compared to other states in the US.”

Though the exact causes of ALS are unknown, Drs. Feldman and Goutman’s research published earlier this year in the Journal of Neurology, Neurosurgery & Psychiatry showed that legacy organochlorine pesticides and other environmental pollutants advance the progression of the neurodegenerative disease. The study measured pesticide levels in blood samples of ALS patients cared for at the U-M Multidisciplinary ALS Clinic.

In March 2019, the Detroit Free Press reported on ALS environmental risk factors: New study: Michigan’s manufacturing legacy may be affecting our health, environment

Other research from Stephen Goutman, M.D., M.S.:

ALS/SURV: a modification of the CAFS statistic. Goutman SA, Brown MB, Cudkowicz M, Atassi N, Feldman EL. Amyotroph Lateral Scler Frontotemporal Degener. 2019 Nov;20(7-8):576-583. doi: 10.1080/21678421.2019.1643375. Epub 2019 Jul 23. PMID: 31334681

Stem cell treatments for amyotrophic lateral sclerosis: a critical overview of early phase trials. Goutman SA, Savelieff MG, Sakowski SA, Feldman EL. Expert Opin Investig Drugs. 2019 Jun;28(6):525-543. doi: 10.1080/13543784.2019.1627324. Epub 2019 Jun 12. Review. Erratum in: Expert Opin Ther Pat. 2019 Jul 4;:1-7. PMID: 31189354

High plasma concentrations of organic pollutants negatively impact survival in amyotrophic lateral sclerosis. Goutman SA, Boss J, Patterson A, Mukherjee B, Batterman S, Feldman EL. J Neurol Neurosurg Psychiatry. 2019 Aug;90(8):907-912. doi: 10.1136/jnnp-2018-319785. Epub 2019 Feb 13. PMID: 30760645

This is an innovative trial that is designed to identify promising new therapies and test these rapidly and simultaneously under one protocol.

“We are thrilled to offer this exciting study at the University of Michigan,” said Stephen Goutman, M.D., M.S., associate director of the U-M ALS Center of Excellence. “This furthers our commitment to developing and testing new therapies for ALS. We have a terrific staff of study coordinators who are prepared to carry out this trial.”

This study is projected to start sometime in 2020. Individuals interested in participating in the trial at Michigan Medicine should contact Jayna Duell, R.N., at 734-936-8776. A complete listing of all 54 sites is available here.

For additional information about the trial, including the drugs to be used, please visit the Mass General website.

About the U-M ALS Center of Excellence: The ALS Center of Excellence is comprised of an active basic science, translational, and clinical research program and the Multidisciplinary ALS Clinic. This structure engages collaboration between physicians, basic scientists, nurses, ancillary providers, and research coordinators all working towards better treatments, an understanding of why a person develops ALS, and ultimately a cure for ALS.

About ALS: Amyotrophic lateral sclerosis (ALS), which is commonly known as Lou Gehrig’s disease, is a progressive fatal neurodegenerative disease that leads to the death of nerve cells in the brain and spinal cord. This in turns causes progressive weakness of voluntary skeletal muscle often leading to death in 2 to 4 years. Current therapies only minimally slow disease and new therapeutic options are critically needed.